The DOI matches its Crossref record; 179 of the 181 references it cites were found in a registry or an index.
Design and development of polymers for gene delivery
Abstract
The lack of safe and efficient gene-delivery methods is a limiting obstacle to human gene therapy. Synthetic gene-delivery agents, although safer than recombinant viruses, generally do not possess the required efficacy. In recent years, a variety of effective polymers have been designed specifically for gene delivery, and much has been learned about their structure-function relationships. With the growing understanding of polymer gene-delivery mechanisms and continued efforts of creative polymer chemists, it is likely that polymer-based gene-delivery systems will become an important tool for human gene therapy.
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“As an alternative, non-viral vectors may offer tantalizing possibility in CRISPR/Cas9 delivery with respect to their low immunogenicity, absence of endogenous virus recombination, less limitation in delivering larger genetic payloads and ease of large-scale production [73–75].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [73]
In the citing paper
“There are two main limitations of non-viral vectors-mediated CRISPR/Cas9 delivery in vivo. The first one is the serum stability of polyplexes after systemic administration [73]. The second one is the relatively low transfection efficiency in the targeted tissues after administration in vivo [76, 77].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [73]
In the citing paper
“Besides, in contrast with viral vectors, cumulative editing efficacy can be obtained after multiple administrations of non-viral vectors-mediated CRISPR/Cas9 delivery owing to limited immune responses [73, 74, 116, 138].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [73]
In the citing paper
“Nanoparticles smaller than 9 nm can passively diffuse through nuclear pore complexes (NPCs), while larger ones requiring the assistance of import proteins (e.g., importins) to actively shuttle them into the nucleus [73].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [73]
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Metadata
- Authors
- Pack, D. W., Hoffman, A. S., Pun, S., & Stayton, P. S.
- Affiliations
- University of Illinois Urbana-Champaign (US)
- University of Washington (US)
- Corresponding author
- Pack, Daniel W.
- Venue
- Nature Reviews Drug Discovery
- Published
- 2005-07-01
- Volume
- 4
- Issue
- 7
- Pages
- 581-593
- ISSN
- 1474-1776 (ISSN-L)
- 1474-1784
- OpenAlex
- W2003414091
- Cited by
- 2,568
- References
- 181
- Citations by year
- 2026: 55 · 2025: 67 · 2024: 94 · 2023: 76 · 2022: 109 · 2021: 122 · 2020: 125 · 2019: 144 · 2018: 159 · 2017: 157 · 2016: 173 · 2015: 205 · 2014: 180 · 2013: 180 · 2012: 188
- FWCI
- 4.89
- Type
- journal article
- Language
- en
- Open access
- closed
- Locations
- Nature Reviews Drug Discovery (publishedVersion)
- PubMed (publishedVersion)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Life Sciences › Pharmacology, Toxicology and Pharmaceutics › Pharmaceutical Science › Advanced Drug Delivery Systems
- Keywords
- gene therapy, structure-function relationship, polymer chemistry, non-viral vectors
- MeSH terms
- Animals, Cyclodextrins, Genetic Vectors, Humans, Imidazoles, Polymers, Drug Design, Genetic Therapy, Gene Transfer Techniques
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